Design and analysis of phase II cancer trials: A review of statistical methods and guidelines for medical researchers

L. Mariani, E. Marubini

Research output: Contribution to journalArticle

Abstract

A number of statistical methods have been proposed in the last three decades for the design and analysis of phase II trials in the oncological field. Herein, a review of these methods is supplied. Practical guidelines for the proper choice of study design and some worked examples are also given. Based on this material, it appears that one-sample studies are likely to yield reliable results in early phase II testing or when the trial is of a comparative nature, provided that an accurate estimate of the response rate is available for standard treatment. Multi-stage designs are useful for reducing, on the average, the number of patients required. In situations different from those cited herein, controlled trials are preferable but require a larger number of patients. This drawback can be lessened through inclusion in the analysis of information from historical controls or the adoption of early stopping rules.

Original languageEnglish
Pages (from-to)61-88
Number of pages28
JournalInternational Statistical Review
Volume64
Issue number1
Publication statusPublished - Apr 1996

Keywords

  • Binary outcomes
  • Cancer chemotherapy
  • Clinical research
  • Phase II trials
  • Sample size

ASJC Scopus subject areas

  • Mathematics(all)
  • Statistics and Probability

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